A BEAT-PCD consensus statement: a core outcome set for pulmonary disease interventions in primary ciliary dyskinesia
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article
Data de publicação
2024
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Editora
EUROPEAN RESPIRATORY SOC JOURNALS LTD
Autores
KOS, Renate
GOUTAKI, Myrofora
KOBBERNAGEL, Helene E.
RUBBO, Bruna
SHOEMARK, Amelia
ALIBERTI, Stefano
ALTENBURG, Josje
ANAGNOSTOPOULOU, Pinelopi
BEYDON, Nicole
Citação
ERJ OPEN RESEARCH, v.10, n.1, article ID 00115-2023, 11p, 2024
Resumo
Background Consistent use of reliable and clinically appropriate outcome measures is a priority for clinical trials, with clear definitions to allow comparability. We aimed to develop a core outcome set (COS) for pulmonary disease interventions in primary ciliary dyskinesia (PCD). Methods A multidisciplinary international PCD expert panel was set up. A list of outcomes was created based on published literature. Using a modified three-round e-Delphi technique, the panel was asked to decide on relevant end-points related to pulmonary disease interventions and how they should be reported. First, inclusion of an outcome in the COS was determined. Second, the minimum information that should be reported per outcome. The third round finalised statements. Consensus was defined as >= 80% agreement among experts. Results During the first round, experts reached consensus on four out of 24 outcomes to be included in the COS. Five additional outcomes were discussed in subsequent rounds for their use in different subsettings. Consensus on standardised methods of reporting for the COS was reached. Spirometry, health-related quality-of-life scores, microbiology and exacerbations were included in the final COS. Conclusion This expert consensus resulted in a COS for clinical trials on pulmonary health among people with PCD.
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Referências
- Amaral MD, 2022, CURR OPIN PHARMACOL, V63, DOI 10.1016/j.coph.2022.102201
- Barbato A, 2009, EUR RESPIR J, V34, P1264, DOI 10.1183/09031936.00176608
- Behan L, 2019, PEDIATR PULM, V54, P2011, DOI 10.1002/ppul.24507
- Behan L, 2017, THORAX, V72, P832, DOI 10.1136/thoraxjnl-2016-209356
- COMET Management Group, The COMET Initiative
- Gahleitner F, 2021, ERJ OPEN RES, V7, DOI 10.1183/23120541.00320-2021
- Gardner Laura E, 2020, BMC Proc, V14, P7, DOI 10.1186/s12919-020-00191-3
- Goutaki M, 2021, EUR RESPIR J, V57, DOI 10.1183/13993003.04601-2020
- Goutaki M, 2020, ERJ OPEN RES, V6, DOI 10.1183/23120541.00237-2019
- Halbeisen FS, 2022, EUR RESPIR J, V60, DOI 10.1183/13993003.01918-2021
- Hannah WB, 2022, LANCET RESP MED, V10, P459, DOI 10.1016/S2213-2600(21)00453-7
- Knowles MR, 2016, CLIN CHEST MED, V37, P449, DOI 10.1016/j.ccm.2016.04.008
- Kobbernagel HE, 2020, LANCET RESP MED, V8, P493, DOI 10.1016/S2213-2600(20)30058-8
- Kuehni CE, 2018, ERS MONOGR, V81, P282, DOI 10.1183/2312508X.10016717
- Kuehni CE, 2017, EUR RESPIR J, V49, DOI 10.1183/13993003.02514-2016
- Lucas JS, 2019, ERJ OPEN RES, V5, DOI 10.1183/23120541.00147-2018
- Lucas JS, 2017, EXPERT REV RESP MED, V11, P779, DOI 10.1080/17476348.2017.1360770
- Marthin JK, 2021, ERJ OPEN RES, V7, DOI 10.1183/23120541.00301-2021
- Marthin JK, 2010, AM J RESP CRIT CARE, V181, P1262, DOI 10.1164/rccm.200811-1731OC
- Mathioudakis AG, 2022, EUR RESPIR J, V59, DOI 10.1183/13993003.02006-2021
- Needham DM, 2017, AM J RESP CRIT CARE, V196, P1122, DOI 10.1164/rccm.201702-0372OC
- Paff T, 2021, INT J MOL SCI, V22, DOI 10.3390/ijms22189834
- Paff T, 2017, EUR RESPIR J, V49, DOI 10.1183/13993003.01770-2016
- Raidt J, 2022, ERJ OPEN RES, V8, DOI 10.1183/23120541.00139-2022
- Smith PG, 2015, Field Trials of Health Interventions: a Toolbox, V3rd, P98
- Wallmeier J, 2020, NAT REV DIS PRIMERS, V6, DOI 10.1038/s41572-020-0209-6
- Zeevenhooven J, 2020, J PEDIATR-US, V221, P115, DOI 10.1016/j.jpeds.2020.02.032